Mareque M, Montesinos P, Font P, Guinea JM, de la Fuente A, Soto J, Oyaguez I, Brockbank J, Iglesias T, Llinares J, Sierra J. Cost-effectiveness analysis of gemtuzumab ozogamicin for first-line treatment of patients with Cd-33 positive acute myeloid leukaemia in Spain. Clinicoecon Outcomes Res. 2021 Apr 22;2021(13):263-77. doi: 10.2147/CEOR.S302097.


OBJECTIVE: To assess the incremental cost–utility ratio (ICUR) of gemtuzumab ozogamicin (GO) + standard of care (SOC) vs SOC alone for treatment of patients with de novo AML from a Spanish Health Service perspective.

METHODS: A cohort state-transition model, with 12 health-states, was used to estimate the lifetime accumulated cost and benefits in terms of quality-adjusted-life-years (QALYs) in AML patients with favourable, intermediate, and unknown cytogenetic profiles. Patient profile was defined based on the ALFA-0701 trial. Therapeutic regimens were defined by 5 haematologists. SOC was assumed to be idarubicin and cytarabine, the combination most used in Spain. QALYs were estimated by applying utilities for the time spent by the cohort in each health-state and utility decrements associated with adverse events (AE). Total cost (€,2020) included drug-acquisition, hematologic stem-cell transplantation, disease management, AE management and end-of-life costs. Unit costs were derived from local databases. All parameters were validated by haematologist. Costs and outcomes were discounted (3%/year).

RESULTS: Higher cost/patient (€ 177,618 vs € 151,434) and greater QALYs (5,70 vs 4,62) were obtained with GO+SOC vs SOC. The ICUR was € 24,203/QALY gained.

CONCLUSION: This simulation suggests that GO + SOC could be a cost-effective option for treatment of patients with de novo AML in first line.

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